Microcap Capricor soars on interim PhII DMD data showing functional benefit for older patients
With the interim analysis for its Phase II Duchenne muscular dystrophy, little Capricor Therapeutics was merely looking for a guidepost, a signal, a trend that the effects seen in an earlier trial can be replicated with a new dosing regimen and delivery method. Instead, the biotech found itself blessed with a basket of data points that seem to buck the natural history trend and separate the drug from placebo in a statistically significant way, more than doubling its tiny stock $CAPR in pre-market trading.
The primary endpoint for the HOPE-2 trial is PUL 2.0, a measure of upper limb function and one of several ways Capricor quantified skeletal muscle improvement. Execs also spotlighted pulmonary and cardiac benefits in the top-line results — factors that are particularly for the patient population that they are targeting, who tend to be older and non-ambulatory.
Consisting of progenitor cells derived from donor hearts, CAP-1002 is designed to release exosomes that kick off a cycle of muscle repair by suppressing inflammation and driving immunomodulation.
Currently, a number of DMD patients take glucocorticosteroids for that purpose, including prednisone and deflazacort (sold in the US by Marathon as Emflaza). There’s also Sarepta’s exon-skipping drug, eteplirsen (Exondys 51), though its benefits have yet to be borne out; Sarepta and a handful of rivals are now in hot pursuit of a supposed one-time cure to replace the missing dystrophin gene in patients.
“While there had been advances in gene therapy, we believe that controlling inflammation over and above what can be done by steroids is important,” CEO Linda Marbán said in a conference call.
Her husband, Eduardo Marbán, had developed the tech at Johns Hopkins.
Data from the small, placebo-controlled study suggest that with a higher dose than the past and delivered intravenously, the therapy is working. At the six-month cut, 6 patients were evaluable in each arm. For those treated with CAP1002 mid-level PUL — think moving elbows and pulling hand to mouth — deteriorated by an average of 0.2 on a 6-point scale, compared to 0.8 on placebo. The p-value was 0.0389.
This and grip strength are the only improvements still statistically significant after six months (there was also a three-month snapshot), but Capricor stressed positive trends throughout.
“I’d like to just remind people that this population of DMD really represents more than half of all DMD patients and there really have not been therapies that have really focused on this population which have just incredibly high burden of disease,” Craig McDonald, the national PI and a professor at UC Davis, said in the call.
On the key issue of safety, Capricor noted that there had only been one serious adverse event among 30 infusions after it put a standard pre-medication regimen in place to mitigate potential immune reactions. Two patients had had immediate immune reactions to the treatment late last year, which prompted the company to put a voluntary hold on the trial.
Armed with these numbers and an RMAT designation from the FDA, Capricor is now looking into a “lean, efficient” pivotal study to position the cell therapy for a BLA — if it can find the cash to do so.
A few months ago it was forced to chop 21 staffers, or half of its workforce, in order to carry on with clinical activities until the end of this year.
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